Boston Children's Hospital

This study tests a new approach to help children on ECMO (a heart-lung machine) recover from heart injury. Doctors will inject the child's own mitochondria (the energy parts of cells) directly into the heart muscle. The goal is to see if this is safe and helps the heart pump bett…

This study looks at whether children with complex medical conditions who are hospitalized for pneumonia can safely stop antibiotics sooner once they are clinically stable. Researchers will track how many families agree to the approach and monitor for any return of symptoms or hos…

This study tests a device called RELiZORB that attaches to a feeding tube and pre-digests fat, making it easier for children with short bowel syndrome to absorb nutrients. The goal is to see if it can safely reduce or eliminate the need for intravenous (IV) nutrition, which can c…

This study tests whether giving more doses of the drug abatacept after a stem cell transplant can better prevent graft-versus-host disease (GVHD), a serious condition where donor cells attack the patient's body. About 160 people receiving a partially matched donor transplant will…

This study looks at whether whole milk or nonfat milk is better for body composition and heart health in children ages 9 to 12 who have a higher BMI. Researchers will measure body fat, lean muscle, and other health markers over one year. The goal is to help guide healthy milk cho…

This study looks at whether certain foods given through a feeding tube can lower bile acid levels in the stomach and saliva of children who have trouble swallowing and are at risk for breathing in stomach contents. About 60 children who get most of their nutrition through a gastr…

This study looks at boys aged 4 to 36 months with one testicle that hasn't dropped into the scrotum and is instead located in the belly. It compares doing one surgery versus two surgeries to move the testicle down. The main goal is to see which approach leads to better testicle s…

This study tests whether a safe fluorescent dye, called indocyanine green (ICG), can help diagnose biliary atresia in infants under 3 months old. The dye is injected and normally appears in the stool if the bile ducts are open. If no glow is seen in the stool, it may indicate a b…

This study looks at two treatments for a specific type of front knee pain in teenage girls who play sports. One group gets a cortisone shot plus physical therapy, the other gets a saline (salt water) shot plus physical therapy. The goal is to see which approach works better for r…

This study compares two ways to manage pain in children aged 3-17 having tonsil surgery. One uses standard opioids, the other uses a non-opioid drug combo. The goal is to see if the opioid-free approach controls pain just as well while reducing side effects like nausea and agitat…

This study explores whether oral ketamine can help control severe pain from sickle-cell crises in adolescents and young adults. Participants receive ketamine every 8 hours for 48 hours alongside standard pain care. The goal is to improve pain relief and reduce reliance on opioids…

This study looks at children born with esophageal atresia, a condition where the esophagus doesn't form properly. After surgery, many develop a narrowing (stricture) that needs balloon dilation to open it up. The study will compare different inflation times to see if a longer hol…

This study tests a brief, virtual therapy for parents of preschool children (ages 2.5 to 4.5 years) who often use screens like tablets or phones to calm or distract their child. The goal is to reduce this 'regulatory screen use' and help children learn to manage their own emotion…

This study looks at whether thicker blenderized tube feeds can reduce stomach symptoms like nausea and bloating in children who rely on feeding tubes. About 40 children will try both thick and thin versions of the feed over 16 weeks to see which works better. The goal is to impro…

This study tests whether a low dose of interleukin-2 (IL-2) is safe and can help control moderate-to-severe Crohn's disease. About 30 people aged 12 to 80 will receive the drug to find the best dose and check for side effects. The goal is to ease symptoms and improve quality of l…

This study looks at whether problems with how the stomach and esophagus move increase the risk of aspiration (food or liquid entering the airway) in children with brain-related disabilities. About 120 children aged 5-21 who rely on feeding tubes and are at high risk for aspiratio…

This study tests whether metformin, a diabetes drug, can reduce pelvic pain in young women with endometriosis. Fourteen participants will take either metformin or a placebo daily for 6 months and track their symptoms. The goal is to see if this non-hormonal approach offers pain r…

This study compares two numbing drugs injected during spinal fusion surgery for teens with scoliosis. One drug lasts longer, and researchers want to see if it reduces the need for strong painkillers like opioids in the first 3 days after surgery. About 128 teens aged 10-17 will t…

This study tests whether using positive language (like "we're going to give you a special sleepy air") instead of standard medical talk helps children aged 5-10 feel less anxious and behave better when they breathe in anesthesia through a mask. About 128 healthy children having s…

This study tests a program designed to help teens and young adults with sickle cell disease build resilience and cope better. Researchers want to see if the program is practical and helpful for participants. The goal is to reduce depression, anxiety, and pain interference.

This study looks at why newborns with severe heart disease are more likely to develop dangerous blood clots after heart surgery. Researchers will collect blood samples from 10 babies to study tiny molecules called microRNAs that may control clotting. The goal is to better underst…

This study is testing a program called CRUSH, which is designed to teach autistic adults about relationships and sexual health. The program includes group sessions and one-on-one coaching. Researchers want to see if the program is practical, well-liked, and shows early signs of h…

This study is testing whether thickening liquids changes how safely children with swallowing problems (dysphagia) can swallow. Researchers will use special tools to measure swallowing in up to 200 children, comparing thin and thickened liquids. The goal is to find out if thicker …

This study tests a new way for families of hospitalized children to report safety concerns using a mobile tool (email, text, or QR code). The goal is to catch medical errors that hospital staff often miss, especially for families who speak languages other than English. About 656 …

This study aims to find the genes that cause strabismus (crossed or misaligned eyes) and nystagmus (involuntary eye shaking). Researchers will collect DNA from families that have at least three members with strabismus, or a child with infantile esotropia or nystagmus. By comparin…

This study follows about 200 people with cystic fibrosis, ages 6 and up, who are starting a new triple therapy (VTD) after experiencing mental health issues or liver injury from a previous drug (ETI). Researchers will closely monitor for any return of mental health symptoms or li…

This study tests new brain imaging tools to measure oxygen and blood flow in children with sickle cell disease. Researchers will compare these scans with standard MRI to see if they can better track how well treatments like gene therapy or transfusions are working. The goal is to…

This study tests a new MRI software that aims to take pictures of the brain much faster than current methods. Researchers want to see if the quick scans are as clear and useful as standard, slower scans for children and adults with low-grade glioma (a type of brain tumor). The st…

This study looks at how blenderized tube feeds (real food pureed and given through a feeding tube) affect acid reflux in children aged 1-21 who have feeding tubes and use standard formula. Researchers want to see if these diets reduce reflux episodes and improve symptoms. The goa…

This study explores how placing young children into foster care and providing caregiver training impacts their thinking, emotions, and brain development. Researchers will follow 220 children in Brazil who are new to the institutional system. The goal is to understand if early int…

This study looks at neutrophils, a type of white blood cell, in children with sepsis (a severe infection). Researchers want to understand how these cells change and may cause lung injury. By comparing sepsis patients to healthy controls, they hope to find clues for future treatme…

This study is testing a new app called OnTrackCF to see if it is easy and helpful for adults with cystic fibrosis (CF) to use. About 60 adults with CF who take a twice-daily medication will try the app for several weeks. Researchers will measure how often people use the app and h…

This study aims to improve fetal MRI imaging by designing special coils and testing new techniques for pregnant women. Researchers will compare the quality of these advanced images to standard fetal MRI and ultrasound. The goal is to get clearer, more reliable images to help doct…

This study looks at how certain immune cells (monocytes) change during heart surgery that uses a heart-lung machine. Researchers will measure these cells and related chemicals in the blood of 100 patients of all ages. The goal is to better understand inflammation and blood clot r…

This study enrolls 10,000 people with rare or undiagnosed genetic conditions, along with their family members, to collect DNA samples and medical information. Researchers use advanced genetic sequencing to find the genes causing these illnesses. The goal is to improve diagnosis a…

This study tests a computer program that helps doctors decide when to remove a breathing tube from children after heart surgery. About 330 children under 12 who have been on a ventilator for at least 48 hours will take part. The goal is to see if the tool shortens the time they n…

This study looks at whether a mobile health program called Bedtime Stories can help caregivers of children ages 5-11 improve their child's sleep. The program sends personalized sleep tips to caregivers. Researchers want to see if the program is easy to use and if it leads to bett…

This study tests a non-invasive light-based device to monitor brain blood flow and oxygen use in premature infants who have had a brain bleed or developed fluid buildup. Researchers will compare these infants to healthy newborns and those with other causes of fluid buildup. The g…

This study looks at how common sedatives and painkillers behave in the bodies of children on ECMO, a life-support machine. Researchers will measure drug levels in 20 children to better understand dosing. The goal is to improve safety and effectiveness, not to test a new treatment…

This study is collecting health information and blood samples from 100 people with two types of hereditary spastic paraplegia (SPG4 and SPG5A). The goal is to create a shared database and biobank to help researchers better understand the disease and prepare for future clinical tr…

This study looks at how the intestines develop normally in premature and full-term babies, and what goes wrong in diseases like necrotizing enterocolitis (NEC). Researchers will collect small tissue samples during surgery or scope procedures, plus blood and stool, to study cells,…

This study aims to find genetic factors that may cause interstitial cystitis (also called bladder pain syndrome), a condition with no known cause or cure. Researchers are collecting blood or saliva samples and medical information from 1,000 people with the condition and their fam…

This study looks at how much clear fluid (like water or juice) teens aged 13-17 can drink 2 hours before an upper GI endoscopy without affecting safety. About 288 participants will be split into four groups, each drinking a different amount. Doctors will measure the fluid left in…

This study looks at whether thickening the milk or formula of infants who have had a brief unexplained event (BRUE) can reduce symptoms and hospital visits. Researchers will follow 250 babies under 1 year old for up to 12 months, comparing those who get thickened feeds to those w…

This study looks for new genetic causes of epilepsy in babies whose seizures start before 12 months old. Researchers will test the babies' DNA and see how a genetic diagnosis affects treatment and family understanding. The goal is to shorten the time to diagnosis and improve care…

This research study is looking for people with congenital myopathies (muscle diseases present from birth) and their family members. The goal is to find the genes responsible for these conditions, which could lead to better diagnosis and treatments in the future. Participants prov…

This study aims to uncover the genetic causes of hereditary spastic paraplegia (HSP), a group of rare nerve diseases that cause progressive muscle stiffness and disability. Researchers will analyze DNA from 200 children and adults with HSP to find gene variants linked to the cond…

This study is creating a shared database of children who have had deep brain stimulation (DBS) for conditions like dystonia, epilepsy, or Tourette syndrome. By collecting information from multiple hospitals, researchers hope to learn which children benefit most, which brain targe…

This study looks at how to better manage blood transfusions for children having heart surgery. Researchers will create a math model to predict the best blood amounts and products to use, and to understand the risk of red blood cell damage. The goal is to make transfusions safer a…

This study looks at the genes of 300 babies who had brain injury around birth (neonatal encephalopathy) to see if genetics play a role in how they recover. Researchers at Boston Children's Hospital will analyze DNA from babies and their parents. The goal is to find genetic causes…

This study tests a nutrition education program for children aged 2-18 who were recently diagnosed with celiac disease. The program focuses on eating naturally gluten-free foods and avoiding highly processed gluten-free products. Researchers want to see if this approach improves b…

This study uses MRI scans to measure how well the placenta is working in pregnancies where the baby is not growing properly (IUGR). The goal is to help doctors decide the safest time to deliver the baby. About 80 pregnant women with IUGR will have MRI scans, and the results will …

This study collects health information and biological samples from up to 700 people under 30 with early-onset hereditary spastic paraplegia (HSP). Researchers hope to better understand how the disease progresses over time. The goal is to create a registry and biorepository to sup…

This study watches 10,000 children with congenital heart disease who need surgery or procedures not on their heart. The goal is to learn what risks they face and what hospital resources they need. Researchers from many hospitals will combine data to create better safety guideline…

This study looks for genes that cause problems with eye movement and certain nerve conditions present from birth. Researchers will study up to 20,000 people with these disorders. The goal is to better understand the causes, not to test a treatment.

This study aims to better understand Chopra-Amiel-Gordon Syndrome (CAGS), a rare genetic disorder. Researchers will follow up to 125 people with confirmed or suspected CAGS over time, collecting medical history, brain scans, genetic data, and quality-of-life information. The goal…

This study looks at how pain signals in the brain and nerve regrowth in the eye change after corneal crosslinking (CXL) for keratoconus. Researchers will use brain scans (fMRI) and special eye microscopes to track healing in 60 people aged 8-35. The goal is to understand pain bet…

This study looks at four biological markers (biomarkers) in 25 children aged 3 to 6 who have both autism and an anxiety disorder. The goal is to see if these markers can help predict how well a child might respond to treatment and measure any changes over time. Parents will fill …

This study is creating a registry of up to 1,000 people with rare lymphatic anomalies (such as lymphatic malformation, Gorham-Stout disease, and CLOVES syndrome). Researchers will collect information from medical records and annual questionnaires to learn how these conditions pro…

This study aims to uncover genetic factors that contribute to epilepsy and related disorders. Researchers at Boston Children's Hospital will analyze DNA from 5000 people with epilepsy to find gene changes that may explain the wide range of symptoms and outcomes. The goal is to be…

This study aims to improve how doctors counsel families when a baby is expected to be born extremely early (between 22 and 25 weeks of pregnancy). Researchers will train doctors in better communication skills and then ask parents about their knowledge, satisfaction, and decision-…

This study is testing whether a saliva (spit) test can accurately show if someone has used cannabis in the past few days, all done from home. Researchers will enroll 200 people aged 18-30 who use cannabis at least once a week. Participants will collect their own saliva samples at…

This study follows up to 55 people with SSADH deficiency, a rare genetic disorder that affects brain function. Researchers will track symptoms, brain activity, and chemical changes over time to understand how the disease progresses. The goal is to find markers that predict severi…

This study tests a virtual genome center (VIGOR) to help sick newborns in community hospitals get expert genetic care without traveling to specialized centers. It will enroll 750 babies with suspected genetic conditions in the NICU, focusing on low-income and minority families. T…

This study is testing a new, non-invasive device that uses light to measure oxygen levels in the brains of newborn babies who may have suffered a brain injury from lack of oxygen at birth. Researchers will compare these measurements with standard tests and follow the babies' deve…

This study looks at how children with intestinal failure feel about their quality of life, and what medical and social factors play a role. Researchers will follow 750 children and their families across the U.S. and Canada for up to 5 years. The goal is to better understand their…

This study looks at whether a non-invasive lung maneuver can help doctors know if a child under general anesthesia needs fluids for low blood pressure. About 48 children having surgery at Boston Children's Hospital will take part. The goal is to avoid unnecessary treatments and i…

This study is a registry that collects information from children and teens (up to age 21) who have spine surgery using robots and special navigation tools. The goal is to see how accurate the robot-placed screws are and to track radiation exposure and patient recovery. No new tre…

This study is creating a registry for people with Shwachman-Diamond Syndrome (SDS) or similar conditions. Researchers will collect medical records and biological samples from up to 5,000 participants to understand how the disease progresses, what complications occur, and how it i…

This study looks at neutrophils—a type of immune cell—in the blood of children with sepsis, a severe infection that can damage organs. Researchers want to understand how these cells change and cause harm, so they can develop better treatments. About 72 children with sepsis and he…

This study looks at children who have both epilepsy (seizures) and movement problems like twitching or trouble controlling muscles. Researchers will collect medical and genetic information from 500 children across many countries. The goal is to learn more about these rare conditi…

This study looks at whether a simple stomach ultrasound can reliably measure how much food or liquid is left in a child's stomach before surgery. Researchers will compare ultrasound results with a standard blood test in 60 children aged 2 to 18. The goal is to find a safer, faste…

This study follows 200 people with a rare genetic epilepsy called EPM1 to track how the disease progresses over time. Researchers will collect medical data and blood or urine samples to build a biobank. No new treatment is being tested, but the information gathered will help desi…